RegulatoryCompany release

FDA grants priority review to uniQure's Huntington's gene therapy AMT-130, with action date of 1 May 2027

🇺🇸 uniQure N.V. (QURE) · Wed 04 Nov 2026

The agency has accepted uniQure's accelerated-approval application for ifezuntirgene inilparvovec, while the UK MHRA has validated a marketing authorisation application for the same product.

uniQure has announced that the US Food and Drug Administration has accepted for priority review its biologics licence application (BLA) for ifezuntirgene inilparvovec, known as AMT-130, in Huntington's disease. The application seeks accelerated approval, and the agency has set a PDUFA action date of 1 May 2027.

The company also said it has aligned with the FDA on a confirmatory trial: a randomised study of roughly 200 patients. Details of the design beyond its approximate size and randomised format were not provided in the information available to VigiEye.

In the UK, the Medicines and Healthcare products Regulatory Agency (MHRA) has validated uniQure's marketing authorisation application for the therapy. Validation means the application has been judged complete enough for formal assessment; it is not a decision on approval.

In its third-quarter update, dated 4 November 2026, uniQure reported cash of $768.4 million. The company expects this to fund operations into 2030.

For context, priority review is an FDA designation that aims to shorten the target review timeline for therapies that could offer significant improvements in treating serious conditions. Accelerated approval allows earlier authorisation based on surrogate or intermediate endpoints, typically with confirmatory evidence required afterwards.

What it means for your team

  • Regulatory teams should note the 1 May 2027 PDUFA date and the agreed confirmatory trial of about 200 randomised patients, which frames the post-approval evidence expectations under accelerated approval.
  • The parallel MHRA validation shows the product is now in formal review in both the US and UK, a point for teams tracking cross-jurisdiction timelines for advanced therapies.
  • Market-access and pharmacovigilance teams in Huntington's disease may wish to begin early planning for a gene therapy launch, including long-term follow-up and evidence generation, though no approval has been granted.

Company's press release (SEC filing): uniQure N.V. ↗

Article written by VigiEye from the official source (uniQure N.V.). Claims are the company's own. AI-assisted; check the source before acting.

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